DoH_Cape_Town_2012-Macklin

Science at the heart of medicine
Ruth Macklin, PhD
Post-trial access to beneficial
products of research
An ethical obligation
| 112/10/2012
Two underlying premises: Purpose of research
• The purpose of biomedical research is to investigate
> Safe, effective diagnostic and therapeutic methods to
benefit future patients
> Safe, effective preventive methods to benefit at-risk
populations
Science at the heart of medicine
| 212/10/2012
Two underlying premises: justice in research
• The principle of justice (as stated in the Belmont Report)
> …whenever research supported by public funds leads to
the development of therapeutic devices and procedures,
justice demands both that these not provide advantages
only to those who can afford them and that such research
should not unduly involve persons from groups unlikely to
be among the beneficiaries of subsequent applications of
the research.
• Two necessary modifications
– Not limited to support by public funds
– Not limited to therapeutic methods
Science at the heart of medicine
| 312/10/2012
Rights-based approach
• International Covenant on Economic, Social, and
Cultural Rights
> 1. The States Parties to the present Covenant recognize
the right of everyone
• (b) To enjoy the benefits of scientific progress and its
applications
> “States Parties” refers to governments that have signed
and ratified the Convention
> Human rights approach also recognizes a role for
non-state actors
Science at the heart of medicine
| 412/10/2012
The Declaration of Helsinki
• In versions before 2000, the DoH made no explicit
mention of post-trial benefits
• In the DoH 2000-2002, two paragraphs addressed post-
trial benefits
> Statement of benefits to the population in Para. 19
• “Medical research is only justified if there is a
reasonable likelihood that the populations in which the
research is carried out stand to benefit from the results
of the research”
– Sufficiently vague to allow a wide range of possible
interpretations
Science at the heart of medicine
| 512/10/2012
DoH 2000-2002
• Explicit statement of benefits to participants in Para. 30
> “At the conclusion of the study, every patient entered into
the study should be assured of access to the best proven
prophylactic, diagnostic and therapeutic methods
identified by the study.”
• Unrealistic?
• Impossible to implement?
• Would deter sponsors from initiating needed research?
• Who should “assure” access?
Science at the heart of medicine
| 612/10/2012
2004 Note of Clarification
• The WMA hereby affirms its position that it is necessary
during the study planning process to identify post-trial
access by study participants to therapeutic, diagnostic,
and therapeutic procedures identified as beneficial in
the study or access to other appropriate care.
• Post-trial arrangements or other care must be described
in the study protocol so the ethical review committee
may consider such arrangements during its review.
Science at the heart of medicine
| 712/10/2012
2008 Para. 33: Weakening the obligation
• At the conclusion of the study, patients entered into the
study are entitled to be informed about the outcome of
the study and to share any benefits that result from it,
for example, access to interventions identified as
beneficial in the study or to other appropriate care or
benefits.
> No clear obligation to provide beneficial interventions
> What other care is appropriate?
> What other benefits are appropriate?
> Who is under an obligation to share the benefits?
> Who should decide?
Science at the heart of medicine
| 812/10/2012
Proposal for strengthening the paragraph
• Version (1) of new paragraph (preferred version)
> “In advance of a clinical trial, sponsors, researchers, and
host-country governments should make provisions for
post-trial access for all participants who still need an
intervention identified as beneficial in the study. All study
participants should be informed about the outcome of the
study.”
• Names the responsible agents
• Includes host governments as agents
– Improvement over CIOMS guideline
• Reverts to essence of 2002 version
Science at the heart of medicine
| 912/10/2012
Proposal (2) for strengthening
• Sponsors, researchers, and host-country governments
should ensure that all participants who still need an
intervention identified as beneficial in a clinical trial have
access to that intervention at the conclusion of the
study. All study participants should be informed about
the outcome of the study.
> Names the responsible agents
> Less specific than proposal 1
> Does not mention when provision should be arranged
Science at the heart of medicine
| 1012/10/2012
Proposal (3) for strengthening
• At the conclusion of a clinical trial, participants in the
experimental and control arms who still need an
intervention identified as beneficial in the study are
entitled to have access to that method. All study
participants should be informed about the outcome of
the study.
> Weakest of three proposals
> Does not identify responsible agents
> Specifies the control arm as potential beneficial method
• Does not require that access to the
experimental product be provided
Science at the heart of medicine
| 1112/10/2012
Conclusions
• Ensuring post-trial access is a shared responsibility
> Burden does not fall on sponsors alone
> Burden does not fall solely on governments in host
countries
> ‘Ensuring access’ does not simply mean ‘paying for
successful products of research’
• Creative means are needed to develop cooperation
between researchers and sponsors, on one hand, and
other sources of funds
– Global Fund
– PEPFAR
– Gates Foundation
Science at the heart of medicine